Safety and efficacy of RNAi therapy for transthyretin amyloidosis

New England Journal of Medicine

Volumn 369, Issue 9, 2013, Pages 819-829

  Coelho, Teresa ^a   Adams, David ^b   Silva, Ana ^a   Lozeron, Pierre ^b   Hawkins, Philip N ^c   Mant, Timothy ^d   Perez, Javier ^a   Chiesa, Joseph ^f   Warrington, Steve ^e   Tranter, Elizabeth ^e   Munisamy, Malathy ^f   Falzone, Rick ^g   Harrop, Jamie ^g   Cehelsky, Jeffrey ^g   Bettencourt, Brian R ^g   Geissler, Mary ^g   Butler, James S ^g   Sehgal, Alfica ^g   Meyers, Rachel E ^g   Chen, Qingmin ^g   Borland, Todd ^g   Hutabarat, Renta M ^g   Clausen, Valerie A ^g   Alvarez, Rene ^g   Fitzgerald, Kevin ^g   Gamba Vitalo, Christina ^g   Nochur, Saraswathy V ^g   Vaishnaw, Akshay K ^g   Sah, Dinah W Y ^g   Gollob, Jared A ^g   Suhr, Ole B ^h   more..

^a HOSPITAL DE SANTO ANTÓNIO   (Portugal)

^b UNIVERSITÉ PARIS SACLAY   (France)

^c ROYAL FREE HOSPITAL   (United Kingdom)

^d IQVIA   (United States)

^e Hammersmith Medicines Research Limited and Trio Medicines Limited   (United Kingdom)

^f 206 Carnegie Center   (United Kingdom)

^g ALNYLAM PHARMACEUTICALS   (United States)

^h UMEÅ UNIVERSITY   (Sweden)

Author keywords

[No Author keywords available]

Indexed keywords

AGENTS AFFECTING METABOLISM; AGENTS AFFECTING PROTEIN METABOLISM; ALN TTR 01; ALN TTR 02; NANOPARTICLE; PLACEBO; PREALBUMIN; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ADULT; AMYLOIDOSIS; ARTICLE; CLINICAL EFFECTIVENESS; CONTROLLED STUDY; DOSE RESPONSE; DRUG EFFICACY; DRUG FORMULATION; DRUG POTENCY; DRUG SAFETY; DRUG TOLERABILITY; ERYTHEMA; FATIGUE; FEMALE; HUMAN; INJECTION SITE REACTION; LOW DRUG DOSE; MAJOR CLINICAL STUDY; MALE; NONHUMAN; OUTCOME ASSESSMENT; PATIENT MONITORING; PHASE 1 CLINICAL TRIAL; PRIMATE; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; RNA INTERFERENCE; SINGLE DRUG DOSE; TRANSTHYRETIN AMYLOIDOSIS;

EID: 84883118140     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1208760     Document Type: Article

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